Evolution of the AMCP Format for Formulary Submissions

DISCLOSURES: No funding was required for this project. The authors are or have been members of the Format Executive Committee.

A MCP's Format for Formulary Submissions is a groundbreaking document describing the evidence required by health care decision makers to support formulary and coverage policy decisions, thus, shaping the dialogue between pharmaceutical and medical device manufacturers and payer pharmacists and physicians and improving their evaluations. Since its adoption by AMCP in October 2000, the AMCP Format has been through 4 major and several minor revisions, maintaining the original vision to create a platform for efficient exchange of scientific information about a pharmaceutical product. Within this framework, manufacturers can share comprehensive clinical and pharmacoeconomic information (including off-label data) with health care decision makers upon their unsolicited request. From its inception, the authors of the AMCP Format have believed that an open dialogue would benefit patients by helping payers and providers make more thorough assessments of emerging products and improve availability of high value treatments to patients that need them.

■■ Conceptual Origins of the Format
Evidence for new drugs is much more available now than in the 1990s, when Professor David Eddy, who directed the Center for Health Policy at Duke University, and others became dissatisfied with traditional methods of clinical decision making. 1 Eddy discussed the application of epidemiologic methods to patient-level decisions in a JAMA series, "Clinical decision making," from 1990 to 1996, 2,3 followed by the long-running series "Users' Guides to the Medical Literature" by Oxman et al. (1993). 4 Forward-thinking managed care pharmacists and academics began incorporating principles of evidence-based medicine in formulary development and clinical practice guidelines. Among these innovators was Jim Carlson at Seattle-based Group Health Cooperative of Puget Sound. 5 As evidence-based medicine gained traction, health policymakers became concerned about information asymmetry between pharmaceutical manufacturers and decision makers in payer and provider organizations. It was difficult or impossible for payers to obtain detailed clinical trial reports and outcomes assessments until well after approval by the U.S. Food and Drug Administration (FDA). Evidence accessible to payers was often limited to manufacturers' product information marketing materials and Pink Sheet summary reports of FDA advisory committee meetings. Without complete high-quality evidence, thorough evaluation by payers was limited.
In 1992, Congress asked the Office of Technology Assessment (OTA)-an office of the U.S. Congress from 1972 to 1995 that provided Congress with objective analysis of complex scientific and technical issues-to evaluate health technologies, including "literature syntheses, outcomes research, cost-effectiveness analysis, practice guidelines development" and other areas. The 1994 OTA report called for evaluating new as well as existing technologies, focusing on patient outcomes rather than intermediate endpoints and making evidence available to decision makers. 6 Subsequently, Neumann et al. (1996) noted that manufacturers were beginning to use pharmacoeconomic data in promotions. 7 Kessler et al. (1994) described "Therapeutic Class Wars-Promotion in a Competitive Marketplace." 8 Publication of the first edition of the textbook Cost Effectiveness in Health and Medicine in 1996 added to the interest in economic evaluation of drugs. 9 The FDA generally did not evaluate peer-reviewed cost-effectiveness literature, even though the industry "had a history of performing pharmacoeconomic studies without scrutiny of their validity." 7 Professor Sean Sullivan at the University of Washington (UW) School of Pharmacy invited Paul Langley from Latrobe University to visit the UW in 1994 to discuss the novel 1991 Australian guidelines for informing drug coverage by the Pharmaceutical Benefits Advisory Committee (PBAC), which incorporated cost-effectiveness analysis. 10 The result was Langley and Sullivan's 1996 seminal article outlining a framework for developing evidence submission guidelines for decision makers in the United States. 11 The challenge was to apply these principles in our complex multipayer and provider environment. In 1994, Regence BlueShield, a large regional health plan in Seattle, whose independent Pharmacy and Therapeutics (P&T) Committee was chaired by Sullivan, created guidelines for review of new and existing drugs, incorporating the framework of Langley and Sullivan.

■■ The Regence Guidelines Project
Implementing the first Regence guidelines, pharmacy staff found that adequate evidence was not available in a timely fashion. Unpublished studies and information on unapproved indications were difficult to obtain, and humanistic and economic data were not routinely provided by industry. Time required for pharmacists to assemble and synthesize the information was excessive. 12 To address these needs, a more practical guidance document was developed by Sullivan and UW postdoctoral fellows Dell Mather and Darren Augenstein, based on approaches being developed in Australia, England, and Canada. This document was approved by Regence in June 1997 and implemented in a 2-year pilot program, the results of which were published in 1999. 12 Regence asked manufacturers to supply their evidence in a comprehensive product dossier "addressing the clinical outcomes and economic consequences of using a proposed product." The dossier contained several sections later included in the AMCP Format: disease description, product description, anticipated place in therapy, clinical studies, economic evaluations, and a disease-based economic model. The success of this pilot project paved the way for development of the AMCP Format.

■■ The AMCP Format Is Launched
Meanwhile, AMCP leadership became interested in the use of dossiers to help payers obtain the necessary information to provide cost-effective quality health care through a publicly accountable process that reduces variation in formulary decisions. 13 They challenged payers to assess outcomes of care, quality, cost-effectiveness, and overall value. Langley was asked to provide input because of his association with the PBAC in Australia. Subsequent discussions with Sullivan and the Regence team led to the AMCP Format Version 1.0, released in October 2000. 14 The Format Executive Committee, a multistakeholder group representing payers, manufacturers, consultants, and academic experts, was appointed to advise AMCP regarding the ongoing maintenance of the Format, with Sullivan as its first chair. The Format Executive Committee has since authored 3 subsequent major versions and several minor revisions that were approved by the Board of Directors. These revisions are summarized in Table 1.
Seeing the potential of this approach, Premera Blue Cross, also in Seattle, incorporated the AMCP Format into its formulary process in May 2001. This provided a solid platform for Premera's biotechnology management initiative in 2005 14  AMCP received considerable feedback after these public sessions. In general, manufacturers felt that the AMCP Format provided an opportunity to convey the value of a product, including more latitude for economic analyses and evidence; however, they expressed concerns about payer reviewers' proficiency, impact on payer relationships, confidentiality of submissions, and the AMCP Format's unsolicited request. They worried that sensitive proprietary information submitted in a dossier (models and unpublished studies) could become publicly available to competitors and might alarm regulatory authorities. 25 Payers were concerned about staff resources and expertise to properly evaluate dossier contents. The Format Executive Committee worked diligently to address these concerns in subsequent revisions.
Clearly, more training was needed. While managed care organizations requested dossiers, most reported using only the clinical evidence and disease background sections, not the economic models. Researchers questioned the relationship between dossier submission and formulary acceptance, [26][27][28][29][30] and a study assessing the quality of dossier economic models found mixed results. 31 Some researchers had misunderstood the dossier's goal of facilitating accurate product assessment through presentation of both positive and negative features thus preventing formulary placement of a low-value product.

■■ Electronic Dossier Delivery
When the AMCP Format began, most dossiers were submitted as printed binders. In 2007, AMCP and Dymaxium, developer of FormularyDecisions, began discussing a web-based eDossier platform that would meet regulatory standards. Dymaxium's Bridget Olson and Allen Lising developed a successful prototype, proactively engaging the FDA to address regulatory concerns. 32

DISCLOSURES
No funding was required for this project. The authors are or have been members of the Format Executive Committee.

ACKNOWLEDGMENTS
The authors thank David Veenstra, Pete Penna, and Dell Mather for reviewing and providing input and Paula Eichenbrenner for sharing many Formatrelated documents from AMCP Foundation records. The authors also thank the various companies that have sponsored Format-related programs and the annual student P&T competition, through which PharmD students learn to evaluate product dossiers and use the information to write formulary monographs. We are especially grateful to those companies that have volunteered product dossiers for use in the competition. large volumes of information. In March 2010, AMCP and Dymaxium launched a pilot eDossier platform with over 200 decision makers. In 2012, use of the eDossier platform was included in the AMCP Foundation P&T Competition requirements. In 2014, the platform added resources including 150,000 evidence links, health technology assessment insights, reviews by the Institute for Clinical and Economic Review, and other decision-maker tools. 33 In 2019, FormularyDecisions was acquired by Xcenda.
■■ Future of the AMCP Format Over 2 decades, the AMCP Format has adapted to many environmental changes, including the advent of biotechnology and specialty drugs, precision therapies, companion diagnostics, real-world evidence, and patient-centered outcomes. The revision process is designed to adapt to future changes, such as those likely to occur following the disruption of the COVID-19 pandemic. The Format Executive Committee has addressed legal and regulatory concerns, clarifying language to increase stakeholder acceptance. Dossiers made the transition from paper to PDF and then to the internet. Today, the AMCP Format remains best practice guidance for transmission of evidence from manufacturers to health care decision makers. Today's payer pharmacists have little time to meet with manufacturers, but they still need increasingly complex information to conduct comprehensive formulary reviews. In a world that demands efficient communication, the AMCP Format delivers.